GemCis Plus PD-1/PD-L1 Inhibitor in Advanced Biliary Tract Cancer: Efficacy and Immune Microenvironment （BTC-IM-1）

Fudan University100 enrolled

Overview

To evaluate the efficacy and immune microenvironment changes in treatment-naïve advanced biliary tract cancer (BTC) patients receiving first-line standard therapy with gemcitabine/cisplatin (GemCis) combined with PD-1/PD-L1 inhibitors.

This is a prospective, non-interventional, observational study evaluating the efficacy and immune microenvironment changes in treatment-naïve advanced biliary tract cancer (BTC) patients receiving first-line standard therapy with gemcitabine/cisplatin (GemCis) combined with PD-1/PD-L1 inhibitors. The primary endpoint is objective response rate (ORR), with secondary endpoints including disease control rate (DCR), duration of response (DOR), time to response (TTR), progression-free survival (PFS), overall survival (OS), and immune profiling of tumor tissue and peripheral blood before and after treatment.

Study Type

OBSERVATIONAL

Enrollment

100

Conditions

Advanced Biliary Tract Cancer

Interventions

GemCis plus PD-1/PD-L1 inhibitorDRUG

Gemcitabine: 1000 mg/m², intravenous infusion, on Day 1 and Day 8 Cisplatin: 25 mg/m², intravenous infusion, on Day 1 and Day 8 PD-1/PD-L1 inhibitor: An approved agent (e.g., Pembrolizumab, Nivolumab, Atezolizumab, etc.) will be selected based on clinical practice and administered at standard doses and schedules. Each treatment cycle lasts 21 days, continuing until disease progression, unacceptable toxicity, or patient/physician decision to discontinue.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Age ≥18 years. * Histologically confirmed unresectable/metastatic cholangiocarcinoma (intrahepatic, extrahepatic, or gallbladder). * No prior systemic anticancer therapy (chemotherapy, targeted therapy, or immunotherapy). * Planned to receive GemCis+PD-1/PD-L1 inhibitor as standard first-line treatment. * ≥1 measurable lesion per RECIST 1.1. * ECOG performance status 0-1. * Adequate organ function: * ANC ≥1.5 × 10⁹/L, platelets ≥100 × 10⁹/L, hemoglobin ≥9 g/dL. * Total bilirubin ≤1.5 × ULN, AST/ALT ≤3 × ULN (≤5 × ULN if liver metastases). * Creatinine ≤1.5 × ULN or CrCl ≥60 mL/min. * Willing to provide archival/fresh tumor tissue and peripheral blood samples. * Signed informed consent. Exclusion Criteria: * Prior systemic therapy. * Active autoimmune disease requiring immunosuppression. * Active infection requiring IV antibiotics. * HIV-positive or active HBV/HCV infection (HBsAg+ with HBV DNA ≥2000 IU/mL; HCV RNA+). * Symptomatic CNS metastases. * Pregnancy/lactation. * Any condition compromising protocol compliance or data interpretation per investigator.

Locations (1)

Zhongshan Hospital, Fudan University

Shanghai, Shanghai Municipality, China

RECRUITING

Outcomes

Primary Outcomes

Overall survival (OS) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1

OS is defined as the time from study treatment to the date of death of the subject, regardless of the cause of death.

Time frame: max 42 months

Secondary Outcomes

Objective Response Rate (ORR) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1

ORR is defined as the percentage of participants who have a confirmed complete response (CR: disappearance of all target lesions) or partial response (PR: at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum of diameters). Responses are according to RECIST 1.1 as assessed by investigator.

Time frame: max 24 months

Disease control rate (DCR) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1

DCR is defined as the proportion of patients with complete response (CR), partial response (PR) and stable disease (SD).

Time frame: max 24 months

Duration of Response (DOR) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1

DOR is defined as the time from first documented complete or partial response until disease progression, death from any cause, or censoring at date of last tumor assessment.

Time frame: max 24 months

Time to Response (TTR) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1

TTR is defined as the time from the start of treatment until the first objective observation of a response (either partial response, PR, or complete response, CR), provided that the response is subsequently confirmed

Time frame: max 24 months

Central Contacts

Peng Wang, MD

CONTACT

86-21-64041990 peng_wang@fudan.edu.cn

Data from ClinicalTrials.gov

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